An age dependent response to hydroxyurea in pediatric sickle cell anemia patients with alpha thalassemia trait

Blood Cells Mol Dis. 2017 Jul:66:19-23. doi: 10.1016/j.bcmd.2017.07.004. Epub 2017 Jul 31.

Abstract

Hydroxyurea (HU) is a key drug therapy for individuals with sickle cell anemia (SCA), yet its clinical and hematologic responses can be variable. Various studies have reported the role of α-thalassemia as one of the most prevalent heritable traits that may modify HU response. We provide data from 62 pediatric and adolescent patients with SCA, 26 with co-inherited α-thalassemia trait. Our data suggest that altered hematologic and clinical responses to HU therapy are noted in adolescent SCA individuals with co-inherited α-thalassemia trait. Adolescent patients who co-inherited α-thalassemia trait had a greater reduction in vaso-occlusive episodes compared to those without α-thalassemia, despite a less robust fetal hemoglobin induction as well as a lower maximum HU dose. This clinical improvement was associated with a lower MCH and higher RBC count. Responses to HU in younger SCA children (ages 5-11years) with co-inherited α-thalassemia trait, compared to those without α-thalassemia trait, did not show any difference in number vaso-occlusive episodes, fetal hemoglobin induction and change in MCH and RBC count.

Keywords: Alpha thalassemia; Hydroxyurea; Sickle cell anemia.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Adolescent
  • Age Factors
  • Anemia, Sickle Cell / complications
  • Anemia, Sickle Cell / drug therapy*
  • Arterial Occlusive Diseases / chemically induced
  • Child
  • Child, Preschool
  • Erythrocyte Count
  • Fetal Hemoglobin / analysis
  • Humans
  • Hydroxyurea / therapeutic use*
  • alpha-Thalassemia / complications*

Substances

  • Fetal Hemoglobin
  • Hydroxyurea